Gene therapy pioneer says the field is behind — and that delivery technology is ‘embarrassing’

STAT November 21, 2019
Rebecca Robbins

CAMBRIDGE, Mass. — Gene therapy pioneer Dr. James Wilson is disappointed by the progress in his field — and expects current therapies and technologies to be soon surpassed by new approaches.

“In five years, when we look back on the way we’re executing on gene therapy now, we’re going to realize that things are going to be very different,” Wilson said at the STAT Summit on Thursday. “The way in which we’re going to treat Duchenne muscular dystrophy, potentially cure it, is not the way in which it’s being evaluated in the clinic now.”

Wilson, the director of the gene therapy program at the University of Pennsylvania’s medical school, knows a thing or two about disappointment. In 1999, Wilson ran...

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